Preferred Label : Ranosidenib;
NCIt synonyms : IDH1/IDH2 Inhibitor HMPL-306; Isocitrate Dehydrogenase 1/Isocitrate Dehydrogenase 2 Dual Inhibitor HMPL-306; IDH1/IDH2 Dual Inhibitor HMPL-306;
NCIt definition : An orally bioavailable inhibitor of mutated forms of both isocitrate dehydrogenase
type 1 (IDH1, IDH1 [NADP ] soluble) in the cytoplasm and type 2 (IDH2, isocitrate
dehydrogenase [NADP ], mitochondrial) in the mitochondria, with potential antineoplastic
activity. Upon administration, ranosidenib specifically targets and inhibits mutant
forms of IDH1 and IDH2, thereby inhibiting the formation of the oncometabolite 2-hydroxyglutarate
(2HG) from alpha-ketoglutarate (a-KG). This prevents 2HG-mediated signaling and leads
to both an induction of cellular differentiation and an inhibition of cellular proliferation
in tumor cells expressing IDH mutations. IDH1 and 2, metabolic enzymes that catalyze
the conversion of isocitrate into a-KG, play key roles in energy production and are
mutated in a variety of cancer cell types. Mutant forms of IDH1 and 2 catalyze the
formation of 2HG and drive cancer growth by blocking cellular differentiation and
inducing cellular proliferation.;
UNII : PC64OXS2C2;
CAS number : 2301974-60-5;
Molecule name : HMPL-306; HMPL 306;
NCI Metathesaurus CUI : CL1412928;
Origin ID : C175602;
UMLS CUI : C5446653;
Semantic type(s)
- concept_is_in_subset
- has_target
